Slide 1: Results for Phase 1b/2a Study of ATYR1923 in Pulmonary Sarcoidosis
A New Path to Medicine Results for Phase 1b/2a Study of ATYR1923 in Pulmonary Sarcoidosis SEPTEMBER 13, 2021 Exhibit 99.2
Slide 2: Forward Looking Statements
Forward Looking Statements The following slides and any accompanying oral presentation contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “may,” “might,” “will,” “should,” “expect,”
Slide 3: Phase 1b/2a Data Supports ATYR1923 Proof-of-Concept and Clinical Advancement
Phase 1b/2a Data Supports ATYR1923 Proof-of-Concept and Clinical Advancement The trial met its primary endpoint: ATYR1923 was safe and well-tolerated 58% overall steroid reduction from baseline and 22% relative reduction in steroid utilization post taper in the 5.0 mg/kg treatment group 33% of patie
Slide 4: Trial Design
Trial Design 4 FVC = Forced Vital Capacity; PFTs = Pulmonary Function Tests
Slide 5: Study Schema
Study Schema 5 (1) subtherapeutic dose 0 4 8 12 16 20 24 24-week Study Period Week ATYR1923 Placebo 5 mg OCS(1) Steroid taper every 2 weeks Primary endpoint If stable on 5 mg, option to completely titrate off steroids ≥10 mg OCS Steroid dose
Slide 6: Baseline Demographics and Disease Characteristics Generally Balanced
Baseline Demographics and Disease Characteristics Generally Balanced 6 Tables 14.1.2.1.1; 14.1.2.2.1; 14.1.2.3.1; 14.1.2.5.1, 14.2.7.1; 14.2.8.1
Slide 7: Monthly Dosing of ATYR1923 was Safe and Well-Tolerated
Monthly Dosing of ATYR1923 was Safe and Well-Tolerated No new or unexpected findings with repeat dosing No dose-response relationship observed Most frequent AEs were consistent with underlying disease: Cough, fatigue, wheezing No signal of immunogenicity No drug related SAEs No deaths 7 Safety popul
Slide 8: Dose-dependent Reduction in Steroid Utilization Compared with Placebo
Dose-dependent Reduction in Steroid Utilization Compared with Placebo 58% overall steroid reduction from baseline and 22% relative reduction compared to placebo in steroid utilization post taper in the 5.0 mg/kg treatment group 33% of patients able to taper off of steroids completely in 5.0 mg/kg tr
Slide 9: Dose-dependent Improvement in FVC % Predicted Compared with Placebo
Dose-dependent Improvement in FVC % Predicted Compared with Placebo 9 Table 14.2.8.1; Differences represent from MMRM adjusting for baseline FVC % predicted, mean (95% CI) FVC % predicted adjusted mean at week 24 in the placebo arm = 76.8%
Slide 10: Dose-dependent Clinically Meaningful Symptom Improvements Compared with Placebo
Dose-dependent Clinically Meaningful Symptom Improvements Compared with Placebo 10 *p<0.05 on difference between adjusted means from MMRM; Pbo = Placebo MCIDs: TDI - Witek 2003; LCQ – Raj 2009; FAS - de Kleijin 2011 (Negative score is better for Fatigue); KSQ Lung – Baughman 2021; KSQ Lung – Baughma
Slide 11: Phase 1b/2a Data Supports ATYR1923 Proof-of-Concept and Clinical Advancement
Phase 1b/2a Data Supports ATYR1923 Proof-of-Concept and Clinical Advancement ATYR1923 was safe and well-tolerated Strong suggestion of efficacy: Dose-response and consistent positive trends across key efficacy endpoints and multiple analysis populations Clinically meaningful symptom improvements in
Slide 12: ILD Market Opportunity
ILD Market Opportunity 12 Lederer, Martinez. NEJM 2018 All ILDs individually have potential for orphan status aTyr estimates for ATYR1923 in Pulmonary Sarcoidosis, CHP, CTD-ILD; excludes IPF >200 types of ILD: 4 major types comprise 80% of patients IPF is the archetypal fibrotic lung disease, but fi
Slide 13: Q&A
Q&A